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What Is The Status of Raptor Pharmaceutical’s Orphan Drug Procysbi’s FDA PDUFA Date ?

December 26, 2012

Raptor Pharmaceutical announces on December 21, 2012, that orphan drug Procysbi’s (Cysteamine Bitartrate/RP103) FDA PDUFA date is extended by 3 months from January 30, 2013 to April 30, 2013.  Procysbi is for the treatment of Nephropathic Cystinosis. The FDA extension is to provide more time for the FDA to complete the review of information submitted by the company within 3 months of the original PDUFA date of January 30, 2013. No additional studies are requested. Raptor Pharmaceutical currently has no FDA approved drugs.

A 3-month delay doesn’t necessarily have any implication for the FDA’s final decision about Procysbi.   Case in point is NPS Pharmaceuticals’ orphan drug Gattex (Teduglutide), which originally had a PDUFA date of September 30, 2012,  and was then given a 3-month delay to D er 30, 2012. Gattex was just approved of on December 21, 2012.

The drug is estimated to cost approximately $350,000/patient/year based on the pricing of orphan drugs targeting similar population sizes.

Kimberley Lee, a Senior Research Analyst at ThinkEquity, believes that “given significant drawbacks and side effects with Cystagon (current Standard of Care (SOC) from Mylan Inc. & has FDA orphan drug designation) … RP103 would gain market share rapidly upon approval …”. Ms. Lee also says that RP103 has an easy-to-use formulation for the patient, convenient dosing schedule, lower pill burden, and a better side effect profile than Cystagon. Cystagon has a 6-hour dosing schedule and has side effects such as vomiting, body odor, and nausea. On the other hand, RP103 has a 12-hour dosing schedule and less side effects.

On December 20, 2012, Raptor Pharmaceutical signs a $50 million loan agreement with HealthCare Royalty Partners to help fund the commercialization of Procysbi and to also advance the company’s development programs. Under the agreement, Raptor Pharmaceutical receives $25 million at closing and an additional $25 million when the FDA approves Procysbi. Christopher Starr, Raptor Pharmaceutical’s CEO, states that, “This loan agreement with HC Royalty further validates the growing value of Procysbi and Raptor’s pipeline”.

Facts About Nephropathic Cystinosis

1) Inherited lysosomal storage disease causing “systemic toxic cystine accumulation which leads to progressive and irreversible tissue damage and multi-organ failure”

2) Usually diagnosed in infancy; usually kidney transplant by age 10 if untreated

3) Mylan Inc’s orphan drug Cystagon (Cysteamine) is the current Standard of Care (SOC)

4) Approximate WorldWide (WW) patient population of 2,000 – including 500 in US & 800 in EU.

Facts About Procysbi/RP103 (Cysteamine Bitartrate)

1) Receives FDA Orphan Drug Designation (ODD) on October 24, 2006

2) Delayed-release (DR) “microbead” capsule of Cysteamine Bitartrate

3) Also in clinical trials for indications of Huntington’s Disease (HD).


Cystinosis Foundation

Cystinosis  Research Foundation (Natalie’s Wish)

Cystinosis Research Network December 26, 2012 article titled, “Raptor Pharmaceutical: FDA Delay is a Near-Term Negative, But Immaterial Overall”

Raptor Pharmaceutical Pipeline

Raptor Pharmaceutical July 2012 Presentation at JMP Healthcare Conference December 26, 2012 article titled, “3 Compelling Biopharma stocks for 2013”

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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